- Diabetic retinopathy is a
complication of long standing diabetes mellitus, that involves the blood
vessels in the eye.
- With progression, complications
such as retinal detachment and even blindness can occur.
- Novel drug targets a protein
that contributes to retinopathy, and could be a potential treatment.
RUNX1 protein, which is found in the abnormal vessels in the retina could
reduce the severity of blood vessel involvement significantly, according to a preclinical study
Massachusetts Eye and Ear by a team of scientists from Harvard Medical School.
for the Study
- The research team felt that
current treatments for advanced eye disease in diabetes, though effective,
can have complications.
- They hope to make their drug
safe and simple so that it can be self-administered by the patient, thus
avoiding injections and other invasive procedures involved in current
forms of treatment.
- In addition, other diseases
showing abnormal blood vessel growth in the eye such as wet age-related macular degeneration,
cancer and retinopathy of prematurity can also be treated by their
and Findings of the Study
current study is still in the nascent preclinical stage.
- The study team found the occurrence of RUNX1 protein, which has been found to promote angiogenesis
(new blood vessel formation) in the blood vessels affected by diabetic retinopathy, but absent in the normal blood vessels.
- Inhibition of RUNX1 protein by a novel small-molecule drug showed a 50
percent reduction in the severity of retinal blood vessel involvement
in preclinical models.
is Diabetic Retinopathy?
long standing diabetes, the blood vessels of the retina in the eye become
affected. This is referred to as diabetic retinopathy. With progression of the
disease, there is an abnormal increase
in the number of blood vessels
in the retina, referred to as proliferative diabetic retinopathy (PDR).
PDR can cause serious complications
such as clouding of vision, increase in the intraocular pressure
(glaucoma), retinal detachment
and ultimately blindness. In
fact diabetic retinopathy is a leading cause of blindness in the elderly.
‘Novel drug therapy that inhibits activity of RUNX1 protein in the eye could address diabetic retinopathy as well as other similar conditions.’
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How The New Drug Hopes To Score Over Current
Treatments for PDR
- Current treatments for
proliferative diabetic retinopathy (PDR) involve laser treatments and injections that target a growth factor, the
vascular endothelial growth factor (VEGF). In many cases, the eye
injections have to be repeated as often as once a month.
- These treatments are effective
but complications such as retinal detachment and bleeding have been
- According to the authors of
this research, the drug used to
inhibit RUX-1 protein is a small molecule that could cross biological
barriers independently, overcoming the need for painful and frequent
- In fact the treatment could be
administered by the patient himself.
the words of co-corresponding authoráJoseph Arboleda-Velasquez, assistant
professor of ophthalmology at Harvard Medical School and assistant scientist at
Schepens Eye Research Institute of Mass. Eye and Ear, "Our study opens the door
for new modalities of treatment based on small molecules that could cross
biological barriers on their own. Such a treatment could be self-administered by patients and eliminate
the need for intravitreal injections
Future Research Plans
- The research team hopes to determine whether RUX-1 inhibition can target the disease process much
earlier even before the condition becomes clinically apparent.
- Future research plans include determining whether the drug can be
delivered as topical eye
drops rather than injections making the treatment safe, simple and
- Further, they wish to explore
the relationship between RUX-1 and VEGF, since both appear to play a
role in abnormal new blood vessel growth.
the research work does manage to develop a drug that can be topically
administered to treat and probably even prevent PDR, a dreaded complication of
long standing diabetes, it would indeed be a path breaking achievement in the
management of retinopathy.
the words of áco-corresponding authoráLeo Kim, assistant professor of
ophthalmology at Harvard
Medical School and a retina surgeon at Mass. Eye and Ear,
"We're hopeful that we may have an opportunity to change the treatment paradigm
for these conditions. Instead of treating patients after these abnormal blood
vessels form in the eye, we may be able to give patients eye drops or systemic
medications that prevent their development in the first place."
- Researchers identify new target for abnormal blood vessel growth in the eyes - (http://www.masseyeandear.org/news/press-releases/2017/04/researchers-identify-new-target-abnormal-blood-vessel-growth-eyes)
- Diabetic Retinopathy - (http://www.aoa.org/patients-and-public/eye-and-vision-problems/glossary-of-eye-and-vision-conditions/diabetic-retinopathy?sso=y)